Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis

ObjectivesThis systematic review and meta-analysis aimed to evaluate the efficacy and safety of combination of Phosphatidylinositol 3-kinase (PI3K) inhibitors and fulvestrant in patients with advanced breast cancer (ABC) who are hormone receptor-positive (HR+) and human epidermal growth factor recep...

Full description

Saved in:
Bibliographic Details
Main Authors: Xuefeng Li, Hongxian Wang, Shuhui Lin, Tianwen Chen
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-06-01
Series:Frontiers in Oncology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fonc.2025.1556978/full
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850236671481610240
author Xuefeng Li
Hongxian Wang
Shuhui Lin
Tianwen Chen
author_facet Xuefeng Li
Hongxian Wang
Shuhui Lin
Tianwen Chen
author_sort Xuefeng Li
collection DOAJ
description ObjectivesThis systematic review and meta-analysis aimed to evaluate the efficacy and safety of combination of Phosphatidylinositol 3-kinase (PI3K) inhibitors and fulvestrant in patients with advanced breast cancer (ABC) who are hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-).MethodsA systematic search was conducted across the PubMed, Cochrane Library, EMBASE databases and major conference websites (ASCO, ESMO, SABCS) to identify randomized controlled trials (RCTs) evaluating the combination of PI3K inhibitors and fulvestrant in the treatment of advanced breast cancer. Two independent reviewers systematically screened the literature, extracted data, and assessed the risk of bias for the included studies based on predefined criteria. Meta-analysis was subsequently performed using R software in accordance with the PRISMA guidelines.ResultsA total of five randomized controlled trials (RCTs) involving 3,011 patients were included. The findings indicated that the combination of PI3K inhibitors and fulvestrant significantly improved progression-free survival (PFS) (HR = 0.74, 95% CI 0.67-0.80, P < 0.0001) and the objective response rate (ORR) (RR = 1.80, 95% CI: 1.39-2.35, P < 0.0001) compared to placebo plus fulvestrant. However, there was no statistically significant difference in clinical benefit rate (CBR) (RR = 1.10, 95% CI: 0.97-1.25, P = 0.1341). Subgroup analysis indicated that the predefined subgroup of PFS based on PIK3CA mutation status assessed by ctDNA was statistically significant (P = 0.0039), whereas the predefined subgroup of PFS based on PIK3CA mutation status assessed by tumor tissue was not statistically significant (P = 0.1514). In terms of adverse events, the incidence of grade ≥3 events was significantly increased in the PI3K inhibitors combined with fulvestrant group (RR=2.11, 95% CI: 1.73-2.58, P<0.0001), particularly hyperglycemia, rash, and transaminitis (ALT).ConclusionThe combination of PI3K inhibitors and fulvestrant significantly improved PFS and ORR in patients with advanced breast cancer. However, substantial dose-limiting toxicities associated with this therapeutic regimen restrict its broader clinical application. In patients with PIK3CA mutations detected on ctDNA analysis, PFS was significantly improved compared to those with wild-type PIK3CA, suggesting that ctDNA-based PIK3CA mutation status may serve as a potential biomarker for treatment response.Systematic review registrationPROSPERO, identifier CRD42023407466.
format Article
id doaj-art-5d42ea86d378409aa5b057db9be033e3
institution OA Journals
issn 2234-943X
language English
publishDate 2025-06-01
publisher Frontiers Media S.A.
record_format Article
series Frontiers in Oncology
spelling doaj-art-5d42ea86d378409aa5b057db9be033e32025-08-20T02:01:54ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2025-06-011510.3389/fonc.2025.15569781556978Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysisXuefeng Li0Hongxian Wang1Shuhui Lin2Tianwen Chen3Day Surgery Care Unit, Shenzhen Nanshan People’s Hospital, Shenzhen, Guangdong, ChinaDepartment of Breast and Thyroid Surgery, Shenzhen Nanshan People’s Hospital, Shenzhen, Guangdong, ChinaDepartment of Radiation Oncology, Shenzhen Nanshan People’s Hospital, Shenzhen, Guangdong, ChinaDepartment of Breast and Thyroid Surgery, Shenzhen Nanshan People’s Hospital, Shenzhen, Guangdong, ChinaObjectivesThis systematic review and meta-analysis aimed to evaluate the efficacy and safety of combination of Phosphatidylinositol 3-kinase (PI3K) inhibitors and fulvestrant in patients with advanced breast cancer (ABC) who are hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-).MethodsA systematic search was conducted across the PubMed, Cochrane Library, EMBASE databases and major conference websites (ASCO, ESMO, SABCS) to identify randomized controlled trials (RCTs) evaluating the combination of PI3K inhibitors and fulvestrant in the treatment of advanced breast cancer. Two independent reviewers systematically screened the literature, extracted data, and assessed the risk of bias for the included studies based on predefined criteria. Meta-analysis was subsequently performed using R software in accordance with the PRISMA guidelines.ResultsA total of five randomized controlled trials (RCTs) involving 3,011 patients were included. The findings indicated that the combination of PI3K inhibitors and fulvestrant significantly improved progression-free survival (PFS) (HR = 0.74, 95% CI 0.67-0.80, P < 0.0001) and the objective response rate (ORR) (RR = 1.80, 95% CI: 1.39-2.35, P < 0.0001) compared to placebo plus fulvestrant. However, there was no statistically significant difference in clinical benefit rate (CBR) (RR = 1.10, 95% CI: 0.97-1.25, P = 0.1341). Subgroup analysis indicated that the predefined subgroup of PFS based on PIK3CA mutation status assessed by ctDNA was statistically significant (P = 0.0039), whereas the predefined subgroup of PFS based on PIK3CA mutation status assessed by tumor tissue was not statistically significant (P = 0.1514). In terms of adverse events, the incidence of grade ≥3 events was significantly increased in the PI3K inhibitors combined with fulvestrant group (RR=2.11, 95% CI: 1.73-2.58, P<0.0001), particularly hyperglycemia, rash, and transaminitis (ALT).ConclusionThe combination of PI3K inhibitors and fulvestrant significantly improved PFS and ORR in patients with advanced breast cancer. However, substantial dose-limiting toxicities associated with this therapeutic regimen restrict its broader clinical application. In patients with PIK3CA mutations detected on ctDNA analysis, PFS was significantly improved compared to those with wild-type PIK3CA, suggesting that ctDNA-based PIK3CA mutation status may serve as a potential biomarker for treatment response.Systematic review registrationPROSPERO, identifier CRD42023407466.https://www.frontiersin.org/articles/10.3389/fonc.2025.1556978/fullPI3K inhibitorsadvanced breast cancerprogression-free survivalPIK3CA mutationsmeta-analysis
spellingShingle Xuefeng Li
Hongxian Wang
Shuhui Lin
Tianwen Chen
Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
Frontiers in Oncology
PI3K inhibitors
advanced breast cancer
progression-free survival
PIK3CA mutations
meta-analysis
title Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
title_full Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
title_fullStr Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
title_full_unstemmed Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
title_short Efficacy and safety of PI3K inhibitors combined with fulvestrant for HR+/HER2− advanced breast cancer: a systematic review and meta-analysis
title_sort efficacy and safety of pi3k inhibitors combined with fulvestrant for hr her2 advanced breast cancer a systematic review and meta analysis
topic PI3K inhibitors
advanced breast cancer
progression-free survival
PIK3CA mutations
meta-analysis
url https://www.frontiersin.org/articles/10.3389/fonc.2025.1556978/full
work_keys_str_mv AT xuefengli efficacyandsafetyofpi3kinhibitorscombinedwithfulvestrantforhrher2advancedbreastcancerasystematicreviewandmetaanalysis
AT hongxianwang efficacyandsafetyofpi3kinhibitorscombinedwithfulvestrantforhrher2advancedbreastcancerasystematicreviewandmetaanalysis
AT shuhuilin efficacyandsafetyofpi3kinhibitorscombinedwithfulvestrantforhrher2advancedbreastcancerasystematicreviewandmetaanalysis
AT tianwenchen efficacyandsafetyofpi3kinhibitorscombinedwithfulvestrantforhrher2advancedbreastcancerasystematicreviewandmetaanalysis